At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- onasemnogene abeparvovec (biological)
- How long the study runs
- Study runs about 100 months (dates as stated)
- About the drug or intervention
- onasemnogene abeparvovec — biological: Onasemnogene abeparvovec is a non-replicating recombinant adeno-associated virus serotype 9 containing the human survival motor neuron gene under the control of the ytomegalovirus enhancer/chicken β-actin-hybrid promoter.
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Testing a treatment
- Ages
- 0 Years to 100 Years
- Who
- All
- Number of participants
- 20
- Started
- 2022-12-19
- Last checked
- 2026-07
Plain English Summary
What is this study?
- • Testing a new treatment for spinal muscular atrophy (sma)
- • Phase3 - 20 participants
- • This is a global, prospective, multi-center study that is designed to assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial
Who can take part?
- • Ages 0 Years to 100 Years
- • Diagnosed with spinal muscular atrophy (sma)
Where?
- • London - Novartis Investigative Site
- • Newcastle upon Tyne - Novartis Investigative Site
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
This is a global, prospective, multi-center study that is designed to assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial. The assessments of safety and efficacy in Study COAV101A12308 will continue for 5 years after enrollment in this study.
More detail
The study is comprised of a Baseline Visit and 2 Follow-up Periods. For Follow-up Periods 1 and 2, which includes Baseline through Year 5 visits, assessments will be performed at the Investigational site. For the first 2 years (Follow-up Period 1), visits will occur every 6 months. For Years 3 to 5 (Follow-up Period 2) follow-up visits will be conducted annually. All patients will enter the study at the baseline visit and continue for 5 years.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 0 Years - 100 Years
- Who can join: All genders
What the study is looking for
- ✓Participated in an OAV101 clinical trial.
- ✓Written agreement to take part must be obtained before any assessment is performed.
- ✓Patient/Parent/legal guardian willing and able to comply with study procedures.
Who cannot take part
- ✗There are no exclusion criteria for this study.
See the full criteria
Where Is This Study? (2 UK sites)
Novartis Investigative Site
London WC1N 3JH, United Kingdom
Novartis Investigative Site
Newcastle upon Tyne NE1 4LP, United Kingdom